PharmaMar's Yondelis will not be routinely available on the National Health Service for the treatment of women with ovarian cancer after cost regulators ruled that the drug is too expensive in relation to its expected benefit.

In final guidance published this morning, the National Institute for Health and Clinical Excellence said that its Appraisal Committee rejected Yondelis (trabectedin) in combination with pegylated liposomal doxorubicin hydrochloride treatment in ovarian cancer patients because it had concerns over its efficacy compared with standard therapies.

One of the key issues was that PharmaMar failed to submit any clinical evidence comparing the drug against platinum-based chemotherapy, the preferred option for patients whose ovarian cancer has returned, placing a question mark over whether Yondelis was any better at extending survival.

And while there was evidence to suggest that it might be more beneficial to women with ‘partially platinum-sensitive’ ovarian cancer, i.e when the disease returns between six and 12 months after initial platinum chemotherapy, the data was not strong enough to convince the Committee that Yondelis could have a place within the NHS in this subgroup.

The rejection also came despite the offer of a Patient Access Scheme, under which the manufacturer limited the total cost of the drug, as the Committee felt it failed to improve the cost effectiveness enough to justify its use, "given the uncertain benefits it may provide patients,” explained NICE chief executive Sir Andrew Dillon.

Taking all the uncertainties into account, it was estimated that the incremental cost-effectiveness ratio (ICER) for Yondelis could exceed £95,000 per QALY gained for the entire eligible population, and £68,000 for the partially platinum-sensitive subgroup, both of which are far over the normal threshold for NHS use.

Nplate through the door

Elsewhere, the Institute has published final guidance recommending the use of Amgen's Nplate (romiplostim) on the NHS to treat the rare bleeding disorder chronic immune thrombocytopenic purpura (ITP), which affects around 3,000-3,500 patients in the UK.

The cost regulator has endorsed the use of Nplate on the NHS in England and Wales, but only when patients are unresponsive to standard active treatments and rescue therapies, in those who have severe disease, or those at high risk of bleeding requiring frequent courses of rescue therapies.

The offer of a PAS, under which Amgen is offering Nplate at a discounted (confidential) price tag, definitely helped seal the deal, as this helped to bring the drug's ICER to under £20,000 per QALY gained for splenectomised patients and around £30,000 per QALY gained for those with their spleens present, falling within the bounds of what is considered good value to money for the NHS.

In Autumn last year NICE turned away GlaxoSmithKline's rival ITP drug Revolade (eltrombopag) after concluding that  - with a projected cost of £104,100 per QALY for splenectomised patients and £116,750 per QALY for those without - it is far too expensive to be funded on the health service.